IP Library Granted Patent US 9,937,231
Granted Patent B2
US 9,937,231 · App. 14/780,242 · Granted Apr 10, 2018

Methods and agents for treating Alzheimer's disease

Inventors: Rudolph Emile Tanzi (Cohasset, MA); Ana Griciuc (Somerville, MA)
Assignee: THE GENERAL HOSPITAL CORPORATION
A61K38/178A61K31/7004C07K14/4711C07K14/7056C07K16/2851A01K2217/05A01K2217/075A01K2217/15A01K2227/105A01K2267/0312
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Quick Facts
Patent No.
US 9,937,231
App. No.
14/780,242
Granted
Apr 10, 2018
Kind
B2
Abstract

The present disclosure provides compositions and methods useful for treating or preventing diseases or disorders where beta amyloid accumulation or aggregation contributes to the pathology or symptomology of the disease, for example Alzheimer's disease.

Claims (18)

1. A method of treating a neuro-inflammation disorder associated with beta amyloid accumulation in a subject, the method comprising administering to a subject in need thereof an effective amount of an agent that inhibits or reduces the expression or activity of CD33 protein, wherein the agent is a nucleic acid comprising a nucleotide sequence substantially complimentary to at least a part of a nucleic acid encoding the CD33 protein.

2. The method of method of claim 1 , wherein the agent is a nucleic acid and the method comprises administering a vector encoding/expressing the agent to the subject.

3. The method of claim 2 , wherein the vector is a viral vector.

4. The method of claim 3 , wherein the viral vector is an adeno-associated virus (AAV) vector.

5. The method of claim 1 , wherein the neuro-inflammation disorder is Alzheimer's disease (AD) or Lewy body dementia.

6. The method of claim 5 , wherein the neuro-inflammation disorder is Alzheimer's Disease.

7. The method of claim 1 , wherein the agent is siRNA, shRNA, antisense RNA or oligonucleotide, miRNA, anti-microRNA or ribozyme.

8. The method of claim 7 , wherein the agent is siRNA, shRNA or antisense oligonucleotide.

9. The method of claim 8 , wherein the agent is siRNA.

10. The method of claim 1 , wherein the nucleic acid encoding the CD33 protein has a nucleotides sequence of SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 6 or SEQ ID NO: 7.

11. A method of treating a neuro-inflammation disorder associated with beta amyloid accumulation in a subject, the method comprising administering to a subject in need thereof a nucleic acid encoding a CD33 protein, wherein the CD33 protein lacks a sialic acid binding domain.

12. The method of claim 11 , wherein the CD33 protein comprises an amino acid sequence SEQ ID NO: 8.

13. The method of claim 11 , wherein the nucleic acid is a vector.

14. The method of claim 13 , wherein the vector is a viral vector.

15. The method of claim 14 , wherein the viral vector is an AAV vector.

16. The method of claim 11 , wherein the nucleic acid is modified RNA.

17. The method of claim 11 , wherein the neuro-inflammation disorder is Alzheimer's disease (AD) or Lewy body dementia.

18. The method of claim 17 , wherein the neuro-inflammation disorder is Alzheimer's Disease.

Continuity (2)
Provisional Application 61805735 · Mar 27, 2013
Related Publication 20160038566A1 · Feb 11, 2016